Phase 1 Clinical Trial Advances with First Subject Dosed in the E.U. and IND Accepted in the U.S.
SOUTH SAN FRANCISCO, CA, April 6, 2023 – Vega Therapeutics, Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that its clinical trial program for VGA039 has been initiated. The first subject has been dosed in the Phase 1 study of VGA039 (NCT05776069), a multinational clinical trial being conducted following approvals of the Investigational New Drug Application (IND) by the U.S. Food and Drug Administration (FDA) and Clinical Trial Application (CTA) in Europe. The clinical trial program will begin with enrollment of healthy volunteers and expand to include patients with von Willebrand disease (VWD).
VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S — a key co-factor involved in thrombin generation during both initiation and propagation of coagulation — with preclinical data demonstrating efficacy in numerous congenital bleeding disorders, including VWD. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment. VGA039 also has potential to treat additional bleeding disorders, including other factor deficiencies and platelet disorders.
“We are excited to advance VGA039 into the clinic as the first purpose-built antibody therapy for VWD, representing a new approach with the potential to reduce the treatment burden for these patients,” said Gary Patou, MD, Chief Medical Officer of Vega Therapeutics. “It is our goal at Vega to enable better therapies for patients with VWD, and we are excited to have taken the first step by dosing our first subject.”
“I appreciate the dedication of the Vega team in pursuing a novel mechanism of action focusing on VWD. VWD is a bleeding disorder with high treatment burden and unmet medical need for many patients that impacts their health and daily lives,” said Ayesha Zia, MD, Director of Hemostasis Thrombosis Program, University of Texas Southwestern Medical Center.
The single ascending dose portion of the Phase 1 clinical study is designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of VGA039 in healthy volunteers and VWD patients. Vega plans to enroll the Phase 1 clinical trial for VGA039 at clinical trial sites across the U.S. and other countries.
About von Willebrand disease
Von Willebrand disease (VWD) is a bleeding disorder in which the blood does not clot properly. In VWD, defective or low amounts of von Willebrand factor (VWF) lead to insufficient platelet adhesion and unstable clot formation. VWD causes severe bleeding that can damage organs and lead to significant impact on patients’ daily lives. Current treatments are limited and include factor replacement therapies that require frequent intravenous (IV) infusions.
About Vega Therapeutics
Vega Therapeutics is a clinical stage biotechnology company developing novel, first-in-class therapies for rare blood disorders with overlooked patient needs, starting with VWD. The company’s lead product candidate, VGA039, is the first purpose-built antibody therapy for VWD with a novel mechanism of action targeting Protein S. By attenuating Protein S co-factor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 augments and restores thrombin generation during both initiation and propagation of coagulation, addressing a fundamental mechanism of clot formation in VWD. VGA039 has potential to be a universal hemostatic therapy that can treat various bleeding disorders. For more information, please visit www.vega-therapeutics.com and follow us on LinkedIn.
Kathryn Morris, The Yates Network LLC